Chemoprotective Antimalarial Activity of P218 against Plasmodium falciparum: A Randomized, Placebo-Controlled Volunteer Infection Study

Am J Trop Med Hyg. 2021 Feb 8;104(4):1348-1358. doi: 10.4269/ajtmh.20-1165.

Abstract

P218 is a highly selective dihydrofolate reductase inhibitor with potent in vitro activity against pyrimethamine-resistant Plasmodium falciparum. This single-center, randomized, double-blind, placebo-controlled phase Ib study evaluated P218 safety, pharmacokinetics, and chemoprotective efficacy in a P. falciparum sporozoite (PfSPZ) volunteer infection study (VIS). Consecutive dose safety and tolerability were evaluated (cohort 1), with participants receiving two oral doses of P218 1,000 mg 48 hours apart (n = 6), or placebo (n = 2). P218 chemoprotective efficacy was assessed (cohorts 2 and 3) with direct venous inoculation of 3,200 aseptic, cryopreserved PfSPZ (NF54 strain) followed 2 hours later with two P218 doses of 1,000 mg (cohort 2, n = 9) or 100 mg (cohort 3, n = 9) administered 48 hours apart, or placebo (n = 6). Parasitemia was assessed from day 7 using quantitative PCR targeting the var gene acidic terminal sequence (varATS qPCR). By day 28, all participants in cohort 2 (P218 1,000 mg) and 8/9 in cohort 3 (P218 100 mg) were sterilely protected post-PfSPZ VIS, confirming P218 P. falciparum chemoprotective activity. With placebo, all six participants became parasitemic (geometric mean time to positive parasitemia 10.6 days [90% CI: 9.9-11.4]). P218 pharmacokinetics were similar in participants with or without induced infection. Adverse events of any cause occurred in 45.8% (11/24) of participants who received P218 and 50.0% (4/8) following placebo; all were mild/moderate in severity, transient, and self-limiting. There were no clinically relevant changes in laboratory parameters, vital signs, or electrocardiograms. P218 displayed excellent chemoprotective efficacy against P. falciparum with favorable safety and tolerability.

Publication types

  • Clinical Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Animals
  • Antimalarials / administration & dosage*
  • Antimalarials / therapeutic use
  • Cohort Studies
  • Double-Blind Method
  • Female
  • Folic Acid Antagonists / administration & dosage*
  • Folic Acid Antagonists / therapeutic use
  • Human Experimentation
  • Humans
  • Malaria, Falciparum / drug therapy
  • Malaria, Falciparum / prevention & control*
  • Male
  • Mice
  • Mice, Transgenic
  • Middle Aged
  • Parasitemia / drug therapy
  • Placebos / administration & dosage
  • Plasmodium falciparum / drug effects*
  • Random Allocation
  • Sporozoites / drug effects*
  • Volunteers*

Substances

  • Antimalarials
  • Folic Acid Antagonists
  • Placebos